Bayer's billion-dollar Parkinson's disease bet snags FDA fast-track tag

Brain
DA01 is just one part of Bayer’s strategy to tackle the root cause of Parkinson’s disease. It is also working on a gene therapy through its AskBio subsidiary. (Getty Images)

Six weeks after landing in the clinic, Bayer’s stem cell therapy for Parkinson’s disease is getting into the fast lane.

The German pharma picked up the treatment, DA01, by buying Versant Ventures out of their regenerative medicine joint venture BlueRock Therapeutics in a deal worth about $1 billion.

DA01 is a pluripotent stem cell-derived dopaminergic neuron therapy. Translation? The company takes stem cells that can develop into any cell type in the body and engineers them to become neurons that produce the neurotransmitter dopamine. Those neurons are then transplanted into a part of the brain called the putamen. 

RELATED: Bayer's billon-dollar Parkinson's disease bets land in the clinic 

Bayer’s BlueRock unit started a phase 1 study of the treatment in early June and will eventually enroll 10 patients in the U.S. and Canada. The trial will gauge the safety and tolerability of the cell transplantation one year after the procedure. It will also assess cell survival following transplant and motor effects one and two years after transplant. 

A fast-track designation grants a drug’s developer more frequent meetings and written communications with the FDA about the development plan, clinical trial design and the use of biomarkers. It could also open the door to a speedy review or accelerated approval based on surrogate endpoints rather than a demonstrated clinical benefit, as in the case of Biogen’s controversial Alzheimer’s drug, Aduhelm (aducanumab).

“This is another critical step in the BlueRock mission to create authentic cellular medicines to reverse devastating diseases, with the vision of improving the human condition,” said Joachim Fruebis, Ph.D., chief development officer of BlueRock, in a statement.

RELATED: Bayer to pay $240M to buy BlueRock cell therapy joint venture 

DA01 is just one part of Bayer’s strategy to tackle the root cause of Parkinson’s, a neurodegenerative disorder that leads to tremors, stiffness and difficulty with walking, balance and coordination. Current treatments focus on managing symptoms, but their efficacy dwindles over time. 

Besides transplanting dopamine-producing cells into patients’ brains, Bayer is also pursuing a gene therapy for Parkinson’s. It shelled out $2 billion upfront to acquire Asklepios BioPharmaceutical, better known as AskBio, in October 2020, with a promise of $2 billion more in milestones. 

The gene therapy also targets the putamen using an adeno-associated virus to deliver a gene to increase GDNF expression, which has been shown in preclinical studies to boost the regeneration of neurons and motor recovery. The treatment is in a phase 1b study that has enrolled 10 patients since August 2020.

Editor's note: This story has been updated to clarify the source of the treatment.